Trans-splicing ribozyme specific to ApoE4 RNA and use thereof
AssigneeRznomics Inc.
InventorsSeong-Wook Lee, Seung Ryul Han
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Abstract
The present invention relates to a trans-splicing ribozyme specific to Alzheimer's disease, and a use thereof. The trans-splicing ribozyme replaces RNA of genes that cause or increase the risk of Alzheimer's disease with RNA of genes that are beneficial for the treatment of the disease, thereby reducing the expression of the disease-causing genes and increasing the expression of the genes beneficial for the treatment of the disease, and thus can be usefully used to prevent or treat Alzheimer's disease.
Claims14
A trans-splicing ribozyme targeting an apolipoprotein E4 (APOE4) RNA sequence, wherein the trans-splicing ribozyme has a structure of 5′-internal guide sequence (IGS)-ribozyme core region-exon-3′, wherein the IGS region consists of a nucleotide sequence of 6 to 10 nt in length and wherein the IGS region is selected from the group consisting of SEQ ID NO:
- 2,4, and 6.
Dependencies are inferred from the claim text.